New Muscular Dystrophy Treatment Shows Promise in Early Study Led by Children’s National
September 17, 2013

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WASHINGTON, DC — A preclinical study led by researchers at Children’s National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD). The results, published in EMBO Molecular Medicine, show that the drug, VBP15, decreases inflammation and protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone.

Duchenne muscular dystrophy results in severe muscle degeneration and affects approximately 180,000 patients worldwide, mostly children. The current standard treatment uses glucocorticoids, but is limited due to serious side effects leading to fragile bones and suppression of both the immune system and growth.

“These findings, while preliminary, are very promising for advancing the treatment of this disease, which causes disability in so many children worldwide,” said Eric Hoffman, PhD, director of the Center for Genetic Medicine Research at Children’s National. “The study also suggests the potential for new strategies in very early treatment, which could further benefit patients.”

The Children’s National research team also observed that VBP15 inhibits the transcription factor NF-κB, a key cell-signaling molecule found in most cell types that plays a role in inflammation and tissue damage. The team previously found that NF-κB is active in dystrophin-deficient muscle years before the onset of symptoms, suggesting that very early treatment of Duchenne muscular dystrophy patients with VBP15 may prevent or delay the onset of some clinical symptoms.

“VBP15 has an additional property of addressing membrane defects in dystrophic muscle cells,” remarked Kanneboyina Nagaraju, DVM, PhD, the lead author of the study, and principal investigator in the Center for Genetic Medicine at Children’s National. “It is becoming increasingly clear that membrane integrity and repair are crucial factors not only in muscle, but also in cardiovascular, neurodegenerative, and airway disorders. In-creasing pre-clinical data suggest that VBP15 may show efficacy in these other disorders and in many other indications where steroids are used.” Dr. Nagaraju noted.

Initial clinical trials of VBP15 are planned, pending Food and Drug Administration (FDA) review and approval, anticipated in 2014. ReveraGen Biopharma, Inc., the first Children's National private spin-off company, is working closely with Children’s National investigators to translate the pre-clinical findings to DMD patients. ReveraGen Biopharma collaborated with NIH/TRND on VBP15, which was selected through a national competitive process, one of the few inaugural compounds selected through this novel pro-gram.

“The partnership with NIH and support from many others is illustrative of the importance of private-public partnerships that are essential in expediting the development of orphan disease therapeutics,” remarked Edward Connor, MD, CEO, and CMO of ReveraGen Biopharma and Director of Innovation Development at Children’s National. “We are optimistic that the new drug will translate well to patients, and are moving this forward as quickly as possible, while assuring safety.”

About EMBO
EMBO is an organization of more than 1500 leading researchers that promotes excellence in the life sciences. The major goals of the organization are to support talented researchers at all stages of their careers, stimulate the exchange of scientific information, and help build a European research environment where scientists can achieve their best work.

EMBO helps young scientists to advance their research, promote their international reputations and ensure their mobility. Courses, workshops, conferences and scientific journals disseminate the latest research and offer training in techniques to maintain high standards of excellence in research practice. EMBO helps to shape science and research policy by seeking input and feedback from our community and by following closely the trends in science in Europe. For more information: www.embo.org

About ReveraGen Biopharma
Reveragen Biopharma is a privately held drug development company focused on development of anti-inflammatory and personalized medicine products, with an emphasis on neuromuscular disease. Reveragen’s lead compound, VBP15, shows efficacy in animal models of chronic inflammation, including Duchenne muscular dystrophy, inflammatory bowel disease, cystic fibrosis, sickle cell anemia, and asthma. The key advance of VBP15 is the separation and optimization of the different sub-activities of frequently prescribed steroid drugs – transrepression, transactivation, and physicochemical membrane properties. This has enabled the selection of a candidate drug that retains or improves efficacy, but minimizes side effect profiles in pre-clinical models. Reveragen began through technology transfer from Children’s National Medical Center and receives development support from NIH TRND, MDA VP, US Department of Defense CDRMP, Foundation to Eradicate Duchenne, and CureDuchenne.

About Children’s National Health System

Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National is Magnet® designated, and is consistently ranked among the top pediatric hospitals by U.S.News & World Report. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. With a community-based pediatric network, seven regional outpatient centers, an ambulatory surgery center, two emergency rooms, an acute care hospital, and collaborations throughout the region, Children’s National is recognized for its expertise and innovation in pediatric care and as an advocate for all children. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.